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Jain et al. Rare Dis Orphan Drugs J 2024;3:8 https://dx.doi.org/10.20517/rdodj.2023.42 Page 5 of 9

Malaysia 33.94 No formal legislation Allocation of approximately 5.3 million US$ has [37,38]
Health Ministry’s National Strategic Plan for supported 60 patients thus far
Medical Programmes currently under review Private insurance coverage available for some RD
Orphan medicine designation under the purview of
NPRA
Nepal 30.55 No specific legislation on RD Congenital disorders, because of their rarity, have [39,40]
not been addressed at the health policy level
Australia 25.98 2020 National Strategic Action Plan for RDs (the Most medicines subsidized through PBS [41-45]
Action Plan) launched RDs that are rejected by PBS on cost-effectiveness
The Pharmaceutical Benefits Scheme (PBS) grounds are supported by the 1995 Life Saving
enables free medicines for 139 ‘life-saving and Drugs Program
disease prevention’ medicines
Taiwan 23.32 The RD and Orphan Drug Act was approved in The Rare Disease and Orphan Drug Act provides [46-48]
2000. The Act defines RD, and facilitates grants, fast-track approval, protocol assistance, and
research, access of orphan drugs and special market exclusivity
nutritional foods. The Act enables the prevention
and early diagnosis of RDs
Hong Kong 7.35 No RD legislation Clinical diagnosis and assessments, multi- [6,49,50]
disciplinary care and rehabilitation services, and
subsidized drugs are available by the Hospital
Authority, a statutory board that manages all public
hospitals.
Designated Orphan Drugs are covered by the
government's annual financial budget allocated to
the Hospital Authority and complimented by the
Samaritan Fund (51 drugs) and the Community
Care Fund Medical Assistance Programme (37
drugs) since 2021 for means-tested patients
Singapore 5.64 No formal RD legislation or definition of RD. The Rare Disease Fund, a national multi- [51-54]
Criteria for accessing the Rare Disease Fund stakeholder charity fund set up in 2019, has
stipulates rare as < 4/10,000 (i.e., < 1,600 provided financial support to 9 patients (as of
people in Singapore). However, since the fund March 2023) for 7 medicines approved for 5 RDs
only covers ultra-rare diseases currently, the
definition for ultra-rare is < 2 in 50,000 people
(i.e., < 225 people with the condition in
Singapore)
Health Products Act governs orphan drugs as
part of all therapeutic products
New 5.12 No specific policy on rare disorders The Pharmaceutical Management Agency [55-61]
Zealand (Pharmac) decides on behalf of Health New
Zealand (Te Whatu Ora) which medicines and
pharmaceutical products will be subsidized. The
Pharmac Rare Disorders Advisory Committee
evaluates funding applications and makes
recommendations to the Pharmacology and
Therapeutics Advisory Committee (PTAC) and
Pharmac

RD: Rare diseases; CoE: centre of excellence; PLWRD: persons living with a rare disease.

Malaysia in 2023. APARDO has also organized a series of webinars on diverse topics, such as Essential
Medicines and Cell and Gene Therapy. It has also partnered with organizations such as rare diseases
international (RDI) for their initiative, the Global Network for RDs, and regionally, through support
towards the RD Network, previously associated with the Asian Pacific Economic Cooperation (APEC)
program. Over the years, the organization has collected data on patient needs and gaps and contributed to
publications [8,48,62] . As a member of various global organizations such as RDI and the International Rare
Diseases Research Consortium (IRDiRC), APARDO continues to contribute perspectives from the APAC
region with the goal of improving the lives of PLWRD and their families.

Opportunities for strengthened collaborations
The hyper diversity of languages, cultures, paternalistic healthcare systems, and sparsity of developed
patient organizations pose an enormous challenge for effective and efficient exchanges across APAC.
Territorial differences ranging from vast areas (China, India, Australia) to thousands of islands (Indonesia,